Search for Clinical Trial Results
Niemann-Pick Disease Type C1 - 25 Studies Found
Status | Study |
Recruiting |
Study Name: Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C Condition: Neimann-Pick Disease, Type C Date: 2006-06-23 |
Recruiting |
Study Name: Biomarker for Niemann Pick Type C Disease Condition:
Date: 2011-03-01 |
Completed |
Study Name: Tolerability and Safety Study of Recombinant Human Acid Sphingomyelinase in Acid Sphingomyelinase Deficiency Patients Condition: Human Acid Sphingomyelinase Deficiency Date: 2012-11-05 Interventions: Drug: Recombinant human acid sphingomyelinase Administered intravenously every 2 weeks for 26 weeks |
Completed |
Study Name: Miglustat in Niemann-Pick Type C Disease Condition: Niemann-Pick Type C Disease Date: 2007-08-15 Interventions: Drug: miglustat Patients aged 12 years or more randomised to the treatment group will receive 200 mg OGT |
Terminated |
Study Name: Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism Condition:
Interventions: |
Recruiting |
Study Name: Genetic Characterization of Movement Disorders and Dementias Condition:
|
Completed |
Study Name: Application of Miglustat in Patients With Niemann-Pick Type C Condition: Niemann-Pick Disease Type C Date: 2013-01-02 Interventions: Drug: Miglustat miglustat 200mg tid for adult. For children surface area correction will be made. A lowe |